hATTR-PN

Long-term treatment with Onpattro (patisiran) is generally safe and results in a stabilization or lessening of neurologic impairments in people with familial amyloid polyneuropathy (FAP), according to a meta-analysis of published studies. Onpattro was used for a mean of 17.4 months (about 1.5 years) among the 503 patients in the…

Nearly every fifth adult with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP), has renal tubular acidosis (RTA) — a problem that occurs when the kidneys fail to remove acids from the blood into the urine. That’s according to a new study from…

Oct. 26 isn’t just another day; it’s World Amyloidosis Day. As a leader of the New Zealand Amyloidosis Patients Association, I’m excited to use this day to raise awareness in our country and unite with our global community. Your support, especially from those in other countries who follow our group…

People with familial amyloid polyneuropathy (FAP) and other forms of hereditary transthyretin amyloidosis (hATTR) have abnormal blood levels of two proteins, called growth differentiation factor-15 (GDF-15) and uromodulin (Umod), a small study reports. The preliminary findings from 16 patients in Italy suggest GDF-15 and Umod may serve as biomarkers of…

Combination therapy for hereditary transthyretin amyloidosis (hATTR), a group of conditions including familial amyloid polyneuropathy (FAP), appears to be safe, according to a retrospective study, but because its effects “on neurological symptoms could not be assessed,” more studies are needed to confirm its benefits, the researchers wrote. Most of…

Since January, the amyloidosis association I lead here in New Zealand has seen our patient numbers grow like never before, primarily because of newer diagnostic tools and more awareness among doctors about the disease. We’re receiving more emails from diagnosed individuals seeking advice on what to do next,…

Onpattro (patisiran) may help familial amyloid polyneuropathy (FAP) patients who respond poorly to tafamidis meglumine stabilize or have reduced symptoms of nerve damage, called polyneuropathy. The findings are part of of a real-world study in France that analyzed data from 24 patients on tafamidis meglumine, approved as…

The clinical symptoms of hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) — are highly influenced by the type of mutation in the TTR gene a patient has, a review study concludes. The findings indicate that a clinical profile marked mainly by neurologic problems,…

A diagnosis of familial amyloid polyneuropathy (FAP), or the onset of peripheral neuropathy symptoms, may cause patients to worry about missing out on the activities they enjoy. Spending time with family, hosting, cooking, and participating in backyard sports may seem daunting due to neuropathy…

Onpattro (patisiran), an approved treatment for familial amyloid polyneuropathy (FAP), is now listed on Australia’s Pharmaceutical Benefits Scheme (PBS), meaning that eligible patients in the country can access the therapy at a lower cost. The listing specifically covers Onpattro’s use by adults with a genetic FAP diagnosis…